Trial in Children With Growth Failure Due to Early Onset Lysosomal Acid Lipase (LAL) Deficiency/Wolman Disease

This is an open-label, repeat-dose, dose escalation study of SBC-102 (USAN: sebelipase alfa) in children with growth failure due to LAL Deficiency. Eligible subjects will receive once-weekly (qw) infusions of sebelipase alfa for up to 5 years


Study Type: Interventional
Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of SBC-102 (Sebelipase Alfa) in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
Estimated Enrollment: 10
Study Start Date: May 2011
Estimated Study Completion Date: December 2015
Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)

Arms:
- Experimental: Cohort 1 (Weekly IV infusions Dose A of sebelipase alfa)


Related journal: Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease.